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New data reinforce robust efficacy and safety profile of Aspaveli/Empaveli for paroxysmal nocturnal haemoglobinuria at the EHA2022 Congress.- Sobi + Apellis

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Published:11th Jun 2022

Swedish Orphan Biovitrum AB (publ) (Sobi) and Apellis Pharmaceuticals, Inc. reported new analyses of phase III studies that reinforce the robust efficacy and safety profile of Aspaveli/Empaveli (pegcetacoplan) for paroxysmal nocturnal haemoglobinuria (PNH) . The data will be presented at the hybrid European Hematology Association (EHA) Congress in Vienna, Austria.

New analyses demonstrated that treatment with Aspaveli/Empaveli resulted in meaningful improvements in quality of life for treatment-naïve patients and suggested the incidence of thrombosis was comparable to eculizumab, a C5 inhibitor. Additionally, a matching-adjusted indirect comparison (MAIC) showed significant improvements in clinical outcomes in treatment-naïve patients who received Aspaveli/Empaveli compared to C5 inhibitors.

Aspaveli/Empaveli demonstrated meaningful improvements in quality of life in treatment-naïve patients : In an analysis of the PRINCE phase III study, Aspaveli/Empaveli patients who were previously treatment-naïve demonstrated meaningful quality-of-life improvements through 26 weeks, reaching normal or near-normal levels of the general population. These data, which were assessed using multiple measures including the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 scale, will be reported during an oral presentation at the congress.

Results suggested incidence of thrombosis comparable across Aspaveli/Empaveli- and eculizumab-treated patient groups A post-hoc analysis of data from all PNH clinical trials of Aspaveli/Empaveli revealed that there were 1.54 thrombotic events per 100 patient-years for Aspaveli/Empaveli-treated patients compared to 1.77 thrombotic events per 100 patient-years for eculizumab-treated patients prior to entry in the PEGASUS phase III study. Additionally, D-dimer normalisation was comparable across Aspaveli/Empaveli- and eculizumab-treated patient groups in a post-hoc analysis of the phase III studies. D-dimer is a marker of thrombotic risk, one of the most common life-threatening complications of PNH.

Aspaveli/Empaveli demonstrated significant improvements in clinical outcomes versus C5 inhibitors in treatment-naïve patients : Using a MAIC methodology, individual patient data from the PRINCE phase III study were compared to aggregate, published data from the ALXN1210-PNH-301 study, which compared C5 inhibitors ravulizumab and eculizumab in PNH patients who were treatment-naïve. Patients treated with Aspaveli/Empaveli showed significant improvements compared to C5 inhibitors across all key disease measures evaluated, including lactate dehydrogenase normalisation, haemoglobin stabilisation and transfusion avoidance at week 26.

In the absence of a clinical head-to-head study, MAIC is a valid and accepted method for comparative effectiveness research used by health technology assessment bodies across the world. As with other MAIC analyses, matching may not adjust for all confounding factors due to differences inherent in study design and entry criteria. Key limitations include differences in the route of administration, treatment administration schedule and dosing regimen.

Condition: Paroxysmal Nocturnal Haemoglobinuria
Type: drug

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